Pharmacoscopy-guided Clinical Standard-of-care in r/r AML

NCT06138990 | Recruiting Phase 2 DMC

• 1 other identifier: RAPID-01
• Study Type: interventional
• Target: 88
• Locations: 1 country
• Sites: 2

Brief Summary

With an overall survival of below 12 months, the outcome of relapsed/refractory AML (RR AML) is poor, making it a critical challenge to identify effective therapies at this stage. The RAPID-01 trial aims to show for the first time in a randomized and controlled clinical trial that Pharmacoscopy (PCY), a functional precision medicine platform, helps improve clinical standard-of-care treatment selection for patients suffering from relapsed/refractory AML.

Conditions

AML, Adult

Keywords

relapsed / refractory AML; Pharmacoscopy; Functional precision medicine

Outcome Measures

Primary Outcomes (1)

• Complete response (CR) rate at day 28

  day 28

Secondary Outcomes (3)

• Composite response rate (CR+CRh+CRi) at day 28

  day 28

• Rate of patients bridged to allogeneic hematopoietic stem cell transplantation within 3 months post-treatment initiation

  3 months

• Treatment-related mortality within 3 months post-treatment initiation

  3 months

Study Arms (2)

Intervention arm: Pharmacoscopy-guided clinical standard-of-care

EXPERIMENTAL

Patients in the PCY-guided treatment arm will receive one of the clinical standard-of-care treatments suggested by their own PCY results, and confirmed by the treating physician.

Diagnostic Test: Pharmacoscopy

Control arm

ACTIVE COMPARATOR

Patients in the control arm will be treated with clinical standard-of-care therapy for RR AML selected by the physician (physician's choice).

Drug: Clinical standard-of-care (physician's choice)

Interventions

Pharmacoscopy DIAGNOSTIC_TEST

Pharmacoscopy (PCY) is an image-based ex vivo drug testing platform developed by the Snijder lab at the ETH Zurich. PCY measures in the drug response of patient cells from small biopsies using automated microscopy and single-cell image analysis. PCY prioritizes treatments based on their specific efficacy against AML cells, while minimizing toxicity to healthy (non-malignant) cells in the patient biopsy.

Intervention arm: Pharmacoscopy-guided clinical standard-of-care

Clinical standard-of-care (physician's choice) DRUG

Clinical standard-of-care therapy for RR AML selected by the physician (physician's choice).

Control arm

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient with refractory or relapsed AML according to ELN2022 criteria.
• Age 18-70 years.
• Considered to be eligible for intensive chemotherapy.
• Written informed consent.

You may not qualify if:

• Acute promyelocytic leukemia (APL) with PML-RARA or one of the other pathognomonic variant fusion genes/chromosome translocations.
• Blast crisis after chronic myeloid leukemia (CML).
• Considered not eligible for intensive chemotherapy.
• Condition of the patient does not allow to wait for PCY results (patient requires immediate treatment).
• PCY not working / patient sample did not pass the QC steps of PCY.
• Any other serious underlying medical, psychiatric, psychological, familial or geographical condition, which in the judgment of the principal investigator may interfere with the project or affect patient compliance.
• Legal incompetence or Subjects lacking capacity to provide informed consent.
• Participation in a clinical trial with an investigational drug within the 30 days preceding and during the present investigation.

Sponsors & Collaborators

• ETH Zurich: lead
• University of Zurich: collaborator
• Insel Gruppe AG, University Hospital Bern: collaborator

Study Sites (2)

Inselspital Bern

Bern, Canton of Bern, 3010, Switzerland

RECRUITING

University Hospital Zurich

Zurich, Canton of Zurich, 8091, Switzerland

RECRUITING

Related Publications (3)

• Snijder B, Vladimer GI, Krall N, Miura K, Schmolke AS, Kornauth C, Lopez de la Fuente O, Choi HS, van der Kouwe E, Gultekin S, Kazianka L, Bigenzahn JW, Hoermann G, Prutsch N, Merkel O, Ringler A, Sabler M, Jeryczynski G, Mayerhoefer ME, Simonitsch-Klupp I, Ocko K, Felberbauer F, Mullauer L, Prager GW, Korkmaz B, Kenner L, Sperr WR, Kralovics R, Gisslinger H, Valent P, Kubicek S, Jager U, Staber PB, Superti-Furga G. Image-based ex-vivo drug screening for patients with aggressive haematological malignancies: interim results from a single-arm, open-label, pilot study. Lancet Haematol. 2017 Dec;4(12):e595-e606. doi: 10.1016/S2352-3026(17)30208-9. Epub 2017 Nov 15.

  PMID: 29153976 BACKGROUND

• Kornauth C, Pemovska T, Vladimer GI, Bayer G, Bergmann M, Eder S, Eichner R, Erl M, Esterbauer H, Exner R, Felsleitner-Hauer V, Forte M, Gaiger A, Geissler K, Greinix HT, Gstottner W, Hacker M, Hartmann BL, Hauswirth AW, Heinemann T, Heintel D, Hoda MA, Hopfinger G, Jaeger U, Kazianka L, Kenner L, Kiesewetter B, Krall N, Krajnik G, Kubicek S, Le T, Lubowitzki S, Mayerhoefer ME, Menschel E, Merkel O, Miura K, Mullauer L, Neumeister P, Noesslinger T, Ocko K, Ohler L, Panny M, Pichler A, Porpaczy E, Prager GW, Raderer M, Ristl R, Ruckser R, Salamon J, Schiefer AI, Schmolke AS, Schwarzinger I, Selzer E, Sillaber C, Skrabs C, Sperr WR, Srndic I, Thalhammer R, Valent P, van der Kouwe E, Vanura K, Vogt S, Waldstein C, Wolf D, Zielinski CC, Zojer N, Simonitsch-Klupp I, Superti-Furga G, Snijder B, Staber PB. Functional Precision Medicine Provides Clinical Benefit in Advanced Aggressive Hematologic Cancers and Identifies Exceptional Responders. Cancer Discov. 2022 Feb;12(2):372-387. doi: 10.1158/2159-8290.CD-21-0538. Epub 2021 Oct 11.

  PMID: 34635570 BACKGROUND

• Schmid JA, Festl Y, Severin Y, Bacher U, Kronig MN, Snijder B, Pabst T. Efficacy and feasibility of pharmacoscopy-guided treatment for acute myeloid leukemia patients who have exhausted all registered therapeutic options. Haematologica. 2024 Feb 1;109(2):617-621. doi: 10.3324/haematol.2023.283224. No abstract available.

  PMID: 37439341 BACKGROUND

Related Links

• Snijder Lab website

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Alexandre Theocharides, MD PhD

  University of Zurich

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Berend Snijder, PhD

CONTACT

+41 44 633 71 49; bsnijder@ethz.ch

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This is a randomised, controlled, parallel group, superiority interventional trial.

Study Record Dates

• First Submitted: November 14, 2023
• First Posted: November 18, 2023
• Study Start: September 2, 2024
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): June 30, 2026
• Last Updated: May 16, 2025

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: CSR
• Time Frame: De-identified patient data will be made available upon publication of the study results in a peer-reviewed internationally recognized journal.

Locations

CH (2)